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Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy. Journal of comparative effectiveness research Campbell, C., Barohn, R. J., Bertini, E., Chabrol, B., Comi, G. P., Darras, B. T., Finkel, R. S., Flanigan, K. M., Goemans, N., Iannaccone, S. T., Jones, K. J., Kirschner, J., Mah, J. K., Mathews, K. D., McDonald, C. M., Mercuri, E., Nevo, Y., Pereon, Y., Renfroe, J. B., Ryan, M. M., Sampson, J. B., Schara, U., Sejersen, T., Selby, K., Tulinius, M., Vilchez, J. J., Voit, T., Wei, L., Wong, B. L., Elfring, G., Souza, M., McIntosh, J., Trifillis, P., Peltz, S. W., Muntoni, F., PTC124-GD-007-DMD Study Group, ACT DMD Study Group, Clinical Evaluator Training Groups 2020

Abstract

Aim: Assess the totality of efficacy evidence for ataluren inpatients withnonsense mutation Duchenne muscular dystrophy (nmDMD). Materials & methods: Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT) populations and two patient subgroups (baseline 6-min walk distance [6MWD] =300-<400 or <400m). Meta-analyses examined 6MWD change from baseline to week 48. Results: Statistically significant differences in 6MWD change with ataluren versus placebo were observed across all three meta-analyses. Least-squares mean difference (95% CI): ITT (n=342), +17.2 (0.2-34.1) m, p=0.0473; =300-<400m (n=143), +43.9 (18.2-69.6) m, p=0.0008; <400m (n=216), +27.7 (6.4-49.0) m, p=0.0109. Conclusion: These meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMDover 48weeks. Treatment benefit was most evident in patients with a baseline 6MWD =300-<400m (the ambulatory transition phase), thereby informing future trial design.

View details for DOI 10.2217/cer-2020-0095

View details for PubMedID 32851872